Table of Contents

1. Overview.- 2. Approaches to the Development and Use of PROs: A Unified Framework.- 3. An Illustration of the New PRO Measure Development Roadmap Using an Example from Skin Disease.- 4. Determination of Fitness for Purpose: Content Verification by Patients and Experts.- 5. Conventional Methods Meeting Modern Techniques: Factor Analysis Versus Rasch Analysis.- 6. Assessing the Performance of PROs Against Expectation: Psychometric Calibration.- 7. Integrating Patient Reported Outcomes in Clinical Trials, Routine Clinical Practice and Medicines Development Programmes.

About the Author

Dr Paul Kamudoni (PhD MSc.) is a Scientific Associate Director in the Global Evidence and Value Development - R&D department at Merck KgaA in Darmstadt (Germany) where he leads the Centre of Excellence for Patient Reported Outcomes. Dr Kamudoni trained as a Pharmacoeconomist and a Clinical Outcome Measurement Scientist at Cardiff University. In his day to day work, he is involved in the development and validation of PRO measures, and the design and integration of PRO endpoints in clinical trials and other types of studies. He maintains an active research interest in methodological issues in the integration of electronic health records with prospective PRO assessments, and the application of item response theory in PRO measurement among others.Dr Nutjaree Pratheepawanit Johns (PhD PharmD) is an Associate Professor at the Faculty of Pharmaceutical Sciences, and melatonin research group, Khon Kaen University, Thailand. She has held a number of academic positions both in Thailand and abroad, including a fellowship at the Western Institutional Review Board (WIRB) (Olympia, WA, USA). Dr Johns has an expertise in cancer care and quality of life (QoL) research and collaborates with several cancer centres in Northeastern Thailand. She has developed and tested a number of PRO measures including cross-cultural adaptation into the Thai language. Dr Johns is also a principal investigator in many clinical trials investigating the effects of disease or interventions on QoL of cancer patients. She has been a recipient of several prestigious awards including Scholarship for Developing Country, International Society for Quality of Life Research and the Doyle Prize: Postgraduate section, Palliative Medicine Journal.
Professor Sam Salek (PhD RPh FFPM FRPS MCMS FESCP) is Professor of Pharmacoepidemiology at the University of Hertfordshire, Hatfield, visiting Professor at the Estate of Hessen, Germany, and Director of the Institute for Medicines Development, Cardiff, UK. Professor Salek's research focuses mainly on four areas: development and application of PRO measures; drug safety evaluation and pharmacovigilance; pharmacoeconomics and economic evaluation of healthcare; and pharmaceutical regulation & medicines development in the mature and emerging markets. He has published 22 books and 600+ journal articles and abstracts. Professor Salek is a member of five Editorial Boards and an active member of several societies.

Reviews

"This is a very detailed outline of a new process that moves away from the traditional models of outcomes research and quantifies the value in the eyes of the patient, which is frequently lost in traditional outcomes assessment. It is an extraordinarily valuable read for policy makers at all levels." (Vincent F Carr, Doody's Book Reviews, January, 25, 2019)